Massachusetts-based gene therapy company Sarepta Therapeutics and California-based biopharmaceutical company Arrowhead Pharmaceuticals have entered a global licensing and partnership deal focused on developing treatments for rare genetic diseases.

Under the agreement, Arrowhead will receive USD 825 million, consisting of USD 500 million in cash and USD 325 million as an equity investment. Arrowhead will also receive USD 250 million to be paid in equal installments over five years, a potential additional USD 300 million in near-term payments, up to USD 10 billion in milestone payments, and royalties on sales.

The licensing agreement includes four clinical-stage programs, three preclinical candidates, and up to six potential discovery programs. Arrowhead will use its Targeted RNAi Molecule (TRiM) platform to conduct discovery and preclinical development activities. Sarepta will conduct the clinical development and commercialization activities of the resulting programs. The deal is expected to be finalized in early 2025.

Sarepta Therapeutics is a biopharmaceutical company focused on developing genetic medicines for rare diseases. The company primarily focuses on treatments for Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs). Sarepta has developed a multi-platform Precision Genetic Medicine Engine spanning gene therapy, RNA, and gene-editing technologies.